drugset

drugset / Trial / NCT03179631

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631 ↗

Phase 3 Completed 360 enrolled PTC Therapeutics

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Timeline

Start: 2017-07-06
Primary completion: 2022-03-05
Completion: 2023-07-25

Publications

Wu S, Vlodavets D, de Queiroz Campos Araujo AP, Castle J. Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041. J Comp Eff Res. 2026 Apr;15(4):e250184. doi: 10.57264/cer-2025-0184. Epub 2026 Mar 25.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ataluren	Small molecule	10 mg/kg	Oral
Subject	Ataluren	Small molecule	20 mg/kg	Oral

Indications